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Overcoming Rare Diseases with Policy

As we know research costs money – lots of money and especially when it comes to medical research demand drives development. (There are a lot of private companies that develop treatment.) The prevalence of a disease often influences the priorities it has in term of government funding as well. For example, cancer while it falls under the umbrella of one disease, different types of cancer require different treatment to be effective. The National Caner Institute in 2007 spent 572.4 million + dollars on Breast Caner research, whereas Pancreatic Cancer (one of the most aggressive cancers, but less common) was given 73.3 million and Stomach Caner was funded at 13.4 million.*

In many ways it make sense to invest research dollars in the diseases that effect the most people, but when you are personally diagnosed with a less common disease that logic blow right out the door. Now cancer is fairly “high profile? disease (it was just an example), but there are rare diseases that effect many people, yet are still such a small portion of the population that its hard to get anyone to fund research. A rare disease is defined as 5 people per 10,000 in the community contract the disease or as the US explains it fewer than 200,000 affected individuals in the country.

In 1983 we had already realized the dilemma and passed the Orphan Drug law which promoted drug development that would normally be too expensive by giving companies a a tax reduction and extended market exclusivity. This law took what was an average of ten drugs developed per decade to over 1,300 in the two decade that followed.

But there are many more hurdles to jump over. Many insurance companies will not cover orphan drugs or they cover them at different rate because they are defined as “specialty drugs.?

Part of the problem is that it is difficult for researchers to prove statistical significant. For example in Phase III Clinical Trials (that is after the drug has been proven to work in a laboratory and has been deemed safe for people, and then been tested on a larger group in Phase II to see how well the drug works) it is often not possible to test the required number of people (Phase II to is typically 20-300; Phase III is 300-3,000 with 1,000 being the preferred point of statistical significant) since less then that number in a community may have the disease.

In the category of Orphan Drugs there is also the challenge of treating children and giving companies the incentive to invest in research for children at the beginning. For example giving longer market exclusivity but requiring that development must start at the beginning and not at the end of the process.

The University of Minnesota is unique in that is has one of the only Centers for Orphan Drug Research in the world. The Center is active in Orphan Drug research and development, but more uniquely it has a public policy focus. The Center is exploring ways to better promote and deliver on Orphan Drug research. It also aims at training medical professionals to be better at dealing with and treating rare diseases. Again, another reason why it “good? to get sick in Minnesota.

*Statistics on cancer funding from the National Cancer Institute “Funding for Various Research Areas.?

Hubert H. Humphrey Institute of Public Affairs
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